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Huntington's Disease 2000 - Change, Advances, and Good News (CAG)
August 18-20, 2000 - Cambridge, Massachusetts
Prepared by Lisa J. Bain
Abstract
At the Hereditary Disease Foundation sponsored, August, 2000 Huntington's Disease research conference in Cambridge, Massachusetts, "Change, Advances, and Good News (CAG)n," progress was reported in numerous areas yet the unanswered questions continued to outnumber those for which there were clear answers. New or modified models for studying the basic biology of the disease and developing new therapies were described. Research aimed at clarifying the relative importance of proteolysis, aggregation, and chaperone proteins were explored, as were the resulting cellular changes, including alterations in gene expression and transcription, electrophysiology, and apoptosis. While no single mechanism emerged as one that would adequately explain this complex disease, increasing effort is being expended towards the goal of finding a treatment. Several new high-throughput screening methods for compounds were described. At least two agents are already in human clinical trials, and other possible therapeutic approaches are under study, including environmental enrichment, inhibition of aggregation, and various approaches that aim to either correct the genetic defect or eliminate or interfere with the mutant protein.
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